Overcoming the Challenges of Gene Therapy

Researchers determine strategies and considerations for making safe AAV-based gene therapies available.

Despite research efforts and numerous ongoing clinical trials, there are only two FDA-approved adeno-associated virus (AAV) gene therapies available. Transitioning gene therapies from the laboratory bench to the clinic entails multiple considerations, including vector optimization, manufacturing, and safety. In this seminar, Dawn Bowles, Samira Kiani, David Schaffer, and Weidong Xiao will discuss the challenges of developing gene therapies and novel strategies to make AAV gene therapies safer and more effective. 

Topics to be covered

  • Gene therapies to improve cardiac transplant outcomes and minimize the need for immunosuppression
  • New immunoregulatory strategies to address immune responses to gene therapies
  • Enhancing viral gene delivery and manufacturing using directed evolution and genetic selection
  • Understanding the risks associated with AAV genome integration

Thursday, June 29th, 2023 | 1:30 PM - 4:00 PM Eastern Time
This seminar will be available to view live and on demand.


Dawn E. Bowles, PhD

Dawn Bowles, PhD

Assistant professor
Duke University

Samira Kiani, MD, PhD

Samira Kiani, MD, PhD

Associate Professor
University of Pittsburgh

David Schaffer, PhD

David Schaffer, PhD

Hubbard Howe Jr. Distinguished Professor
University of California at Berkeley

Weidong Xiao, PhD

Weidong Xiao, PhD

Indiana University

Supported by